Survey on the treatment and

quality of life of patients

Results of the questionnaire survey

Blood Product Subcommittee, Biologics Committee

European Federation of Pharmaceutical Industries and Associations, Japan (EFPIA Japan)

With the Cooperation of Specified Nonprofit Corporation, PID Tsubasa No Kai

Primary immunodeficiency syndrome (PID)

Summary of the Questionnaire Survey

Results

〈Introduction〉

● About European Federation of Pharmaceutical Manufacturers' Associations, Japan (EFPIA Japan)

Established in April 2002, EFPIA Japan is a leading organization of innovative European pharmaceutical

companies operating in Japan and represents 25 R&D-oriented pharmaceutical companies. It aims to

contribute to medical care and patients in Japan through the early introduction of innovative pharmaceuticals

and vaccines. EFPIA Japan activities are carried out through six committees (Pricing & Economics Committee,

Technical Committee, Corporate Ethics Committee, Intellectual Property and Legal Committee, Biologics

Committee, and Public Relations Committee), which discuss and raise awareness of issues related to the

pharmaceutical industry and make proposals to address them. (Home Page: http://efpia.jp/))

● About Blood Product Subcommittee

Blood Product Subcommittee, together with Vaccine Subcommittee and Biological Products Subcommittee,

belongs to Biologics Committee. Blood Product Subcommittee aims to build a framework for the stable supply of

human plasma-derived products (plasma fraction preparations), which are not replaceable by other pharmaceuticals

and are medically indispensable, and blood-related recombinant products manufactured with world's cutting-edge

technologies, to patients in Japan into the future.

This survey was conducted by the Blood Product Subcommittee in EFPIA Japan with the support of Specified

Nonprofit Corporation, PID Tsukuba No Kai. In order to understand various conditions surrounding patients

with primary immunodeficiency (PID), the survey was conducted in members of PID Tsubasa No Kai and

volunteers from PID patients nationwide and asked questions related to hospital visits and patient quality of

life (QoL). Responses were collected through a web-based survey form and a paper-based survey form. PID Tsubasa

No Kai supported the distribution of the paper-based survey form to its members and PID specialists. Medical

institutions involved in PID treatment across the country also supported the distribution of the paper-based survey

form to PID patients. Without their support, it was difficult to collect a substantial number of responses. Member

companies of Blood Products Subcommittee, EFPIA Japan also distributed a total of more than 3,000 copies of the

survey form to medical institutions.

The questionnaire survey was conducted during the period from July 1, 2017 to January 31, 2017, and

responses were collected from 165 patients or patent family members. The Blood Product Subcommittee hopes

that the results of the survey would serve as a useful reference to patients, their families, healthcare

professionals, and other stakeholders.

As an industry-wide organization, EFPIA Japan would like to utilize the results in supporting activities of patient

groups and healthcare professionals.

After confirming the attributes of the respondents, the survey asked them about the following six major items:

"status of hospital visits, complications, and daily precautions", "treatment", "kindergarten and school life",

"employment", "physical disability certification", and " patient needs for society". The results of the survey are

summarized below.

1. Responses were received from 165 persons, including 69 patients, 93 patient families, and three others

(Fig.1 and 2).

2. The respondents with diseases primarily based on humoral immunodeficiency (81 patients) and primary

phagocytic dysfunction and deficiency (42 patients) accounted for approximately 75% of the entire

respondents (Fig.3).

3. 47.9% of the patients were diagnosed as PID at age one or younger, including 31.5% who were diagnosed before

the age of one, indicating that many of the patients were diagnosed during infancy. However, 16.4% received a

definite diagnosis for the first time at the age of 16 to 60 years (Fig.4).

4. 62.4% of the patients visited a medical institution regularly at least once a month (Fig.5), and mostly visited a

pediatric department (Fig.8). The patients saw a specialist, since PID is a rare intractable disease (Fig.9).

5. More than 43% of the patients spent at least one hour to visit a medical institution (Fig.6). 38.8% of the patients

spent three to six hours in a medical institution and 9.1% spent six hours or more (Fig. 7). This suggested that

many patients spared a day for a hospital visit. Considering the characteristics of PID, the long waiting time in a

Objectives and methods of the survey

Overview of the survey results

2

medical institution increases the risk of infection and exhaustion. Improvement is therefore expected in the system

for patient access to PID care to reduce the patient burden for hospital visits.

6. PID patients who are prone to infections frequently develop various symptoms, even when they receive

appropriate treatment depending on the type of PID (Fig.11).

7. When asked about symptoms of the infectious diseases that the patients contracted in the past five years, it was

revealed that they easily acquired opportunistic infections which healthy persons hardly develop, and tended to

suffer from diseases that often leave aftereffects when repeatedly recurred. Besides, the incidence of respiratory

infections, such as bronchitis and pneumonia, was high. In particular, when the patients developed pneumonia,

the rate of hospitalization was as high as 68% (Fig.12).

These become chronic, making treatment difficult and having a major impact on daily life (Fig.13).

8. Many of the PID patients were in poor physical condition and lived a life with limits. It is expected that the survey

results will help patients with PID improve their awareness of the need for infection prevention and gain

understanding of the community (Fig.14 and 15).

9. 58.5% of the patients received immunoglobulin therapy to prevent infections (Fig.16), and as high as 74.2% of the

patients were aware of their IgG trough levels (Fig.18), indicating improved informed consent and increased

patient awareness about their disease status.

10. However, as mentioned above, the incidence of complications including infections and various symptoms

associated with PID remained high, and it was considered that some patients receiving immunoglobulin

replacement therapy may better consult their physician to see if the trough levels are optimal or if complicated

symptoms can be reduced (Fig.19).

11. PID is designated as an intractable disease by the national government and public assistance is available to have

support for the treatment costs for PID and associated complications. However, 31.4% of the patients had

experience where they could not receive public assistance for the treatment costs for complications associated

with PID (Fig.20). When PID patients visit a medical institution, they often need to be accompanied by their family

member or caregiver, which also imposes a large financial burden, suggesting that these issues put pressure on

their livelihood (Fig.21).

12. The survey investigated the impact of PID on school attendance. 64.3% to 70.4% of the patients were able to

attend nursery school, kindergarten, elementary school, junior high school, or high school for 10 to 12 months in

the year (Fig.22), indicating that many patients faced a severe environment in their school life, where they had to

comply with instructions of their physician and live a life with limits to prevent infection (Fig.23 and 24).

13. 36.1% of the patients who attended high school answered that the disease negatively affected the school

performance and 31.9% answered that they had to compromise on career options (Fig.25 and 26).

14. 59% of the patients aged 18 years and older were employed (Fig.27). This was lower than the 75.8%

employment rate of healthy general people aged 15 to 64 years identified in the National Survey of the

Ministry of Internal Affairs and Communications. These findings suggested that the PID patients faced a

difficulty in employment. 41% of the patients were not employed (Fig.27), but many of them wanted to work

(Fig.29).

15. Lack of understanding among colleagues about PID patients who need to visit a medical institution regularly and

take measures to prevent infections impeded them from working. Overwork may worsen the patient condition

and lead to retirement or absence from work. It was highlighted that it was difficult for PID patients to find a

workplace with a good understanding.

16. Approximately 80% of the patients expected that physical disability certification would help reduce economic

burdens of medical and traveling expenses and 80% expected that the disabled employment system would enable

them to work in an appropriate environment (Fig.32). 51.5% of the patients expected much of patient group

activities to achieve designation of the physical disability certification for PID (Fig.33).

17. The survey showed that because of the frequent occurrence of complications, not only medical expenses but

also related costs for visiting a medical institution were pressing on their lives. Furthermore, the disease affected

the school attendance and employment of the patients. Many patients wanted to work but were in a situation

where they were not able to work. It is much expected that these issues would be shared with healthcare

professionals and the Ministry of Health, Labour and Welfare in order to achieve designation of the physical

disability certification for PID.

3

3

Fig.3 Disease type

<Details of the survey results>

■ Patient

■ Family

■ Friend

■ Other

■ Not answered

0- 2 yrs

■ 3- 5 yrs

■ 6-12 yrs

■ 13-15 yrs

■ 16-20 yrs

■ 21-29 yrs

■ 30-39 yrs

■ 40-49 yrs

■ 50-59 yrs

■ 60 yrs or older

■ No answer

・A total of 165 patients responded; 69 (41.8%) were patients, 93 (56.4%) were patient family members, two (1.2%) were “other”,

and one (0.6%) did not answer.

・93 (56.7%) were aged 0 to 20 years, and 71 (43.3%) were aged 20 years or older, excluding the one who did answer.

X-linked agammaglobulinemia

Unclassifiable immunodeficiency

Hyper-IgM syndrome

IgG subclass deficiency

Selective IgA deficiency 29.VI. Deficiency of specific antibody

Other diseases mainly caused by humoral immunodeficiency

Chronic granulomatosis

Severe congenital neutropenia

Other leukocyte dysfunctions

Other chronic courses of neutropenia

X-linked severe combined immunodeficiency

Adenosine deaminase (ADA) deficiency

Other combined immunodeficiency disorders

Hyper-IgE syndrome

Wiskott-Aldrich (Wiskott-Aldrich) syndrome

Dyskeratosis congenita

Ataxia telangiectasia

X-linked lymphoproliferative syndrome

Other immune dysregulation disorders

Other innate immune anomalies

Other innate complement deficiencies

Other

Unknown

■ Diseases mainly involving humoral immunodeficiency ■ Primary phagocytic insufficiency and deficiency ■ Combined immunodeficiency

■ Characteristic syndrome with immunodeficiency ■ Immune dysregulation ■ Innate immune abnormalities

■ Inherited deficiency of complement system ■ Other ■ Unknown

The disease types of the respondents were similar to those frequently reported in the national survey by the Ministry of Health,

Labour and Welfare. 81 patients (49.1%) had diseases mainly due to humoral immunodeficiency and 42 patients (25.5%) had

primary phagocytic insufficiency and deficiency. These two disease types accounted for 74.5% of the entire respondents.

1. Attributes of the patients who responded

Fig.1 Respondents Fig.2 Age

165 respondents (multiple choice n=190)

165 respondents 165 respondents

(Number of

Responses)

4

■ 0 yr ■ 1 yr ■ 2 yrs ■ 3 yrs ■ 4 yrs ■ 5 yrs ■ 6-15 yrs ■ 16 to 60's yrs ■ Not answered

47.9% of the patients were diagnosed as PID at age one or younger, including 31.5% who were diagnosed before

the age of one, indicating that early diagnosis was quite prevalent. However, 16.4% of the patients received a

definite diagnosis for the first time at the age of aged 16 to 60 years. This may be because some PIDs do not

cause severe symptoms in childhood or develop in adulthood.

Almost once a week

Once every 2 weeks

Once a month

Once every 2 months

Once every 3-4 months

Once every 6 months

Once a year

Multiple medical institutions

Regular visit is not necessary

Others

0% 10% 20% 30% 40% 50%

103 patients (62.4%) visited a medical

institution once a month or more

frequently. 29 patients (17.6%) answered

once a week to once every two weeks.

Such frequent visits were considered to

be due to the treatment of

complications. 30 patients (18.2%)

regularly visited multiple medical

institutions.

3 hrs or more

2 to 3 hrs

1 to 2 hrs

30 mins to 1 hr

Less than 30 mins

Many patients spent less than

one hour to visit a medical

institution. However, 43% of the

patients took more than one

hour to receive the treatment for

PID, a rare intractable disease,

and some spent more than three

hours.

Fig. 6 Travel time to visit a hospital (one-way)

165 respondents (multiple choice n=222)

2. Hospital visits and complications

Fig. 5 Frequency of hospital visits for PID treatment

165 respondents (multiple choice n=177)

Fig. 4 Age when the patients were diagnosed PID 165 respondents

(Number of

responses)

5

(Number of

responses)

5

6 hrs or more and return in a day

5 hrs to 6 hrs

3 to 4 hrs

2 to 3 hrs

1 to 2 hrs

Less than 1 hr

Hospitalization for regular treatment

Other

38.8% of the patients stayed in a medical institution for three to six hours, and 9.1% for six hours or more,

indicating that many patients spared a day for a hospital visit.

■ Pediatrics

■ Internal medicine

■ General medicine

■ Hematology

■ Collagen diseases

■ Other

Of the total respondents in this survey,

while 43.7% were patients 15 years of age

or younger, as many as 61.2% visited

pediatrics. It was considered that patients

15 years of age and older continue to visit

pediatrics, and patients diagnosed with

PID in and after adulthood often visit

pediatrics where many specialists belong

to.

■ Yes (specialist)

■ Collaboration between

specialists and general

physicians

■ No (general physician)

■ I do not know.

■ Other

As PID is a rare intractable disease, the

proportion of the patients who saw a

specialist was very high. Nevertheless, for

11.9% of the patients, PID care was

coordinated between specialists and general

physicians. Such care coordination allows

specialists in university hospitals and general

hospitals (core hospitals) to share with

physicians in the patient’s neighborhood

information about appropriate treatment for

acute symptoms, which may reduce the

various burdens on patients for hospital

visits. Therefore, it is necessary to study and

share cases where effective care

coordination between PID specialists and

general physicians is in place.

Fig.7 Time per visit spent in a hospital

Fig. 9 Whether the patients consulted a PID specialist

Fig. 8 Department the patients consulted for the treatment of PID 165 respondents

165 respondents (multiple choice n=170)

159 respondents

(Number of

responses)

6

Autoim

mune disease

Purulent

lymphadenitis

Herpes (including

herpes zoster)

Periproctiti

Cellulitis

Derm

atitis, etc.

Pyelitis

Enteritis

Sinusitis

Otitis m

edia

Osteom

yelitis

Aspergillus

pneumonia

Pneum

onia

Bronchitis

Abscess

University hospital that I regularly visit

General hospital that I regularly visit

Clinic that I regularly visit

Neighboring general hospital different from a regularly visiting hospital

Neighboring clinic different from a regularly visiting hospital

Other

Many patients had to consult the university hospital or general hospital that they regularly visited and might

spend many hours in the hospital for the treatment of acute symptoms of complications such as infectious

diseases where they might be physically exhausted.

■ 1-2 times

■ 3-5 times

■ 6-10 times

■ 10 times or

more

Pyrexia Coughing Tonsillitis Diarrhea Stomatitis Fatigue Abdominal Arthralgia Vomiting

pain

PID patients are susceptible to a variety of infections on a daily basis. Even they may limit activities to prevent

infections, infections and various symptoms can interfere with their lives.

Complications that occurred in the past five years included diseases affecting various sites of the body, which

healthy people do not usually develop and a high incidence of respiratory infections and enteritis, including upper

respiratory tract infections. The rate of hospitalization (number of hospitalizations/incidence) was as high as 68%

(32/47) in cases of pneumonia, and 40% (10/25) in cases of enteritis. Although the incidence was not high, the rate

of hospitalization for skin disorders related to the underlying disease was high. This indicated that it is important

for PID patients to take measures to prevent infections, receive appropriate treatment, and appropriately manage

drug therapy. Complications should be treated early to prevent deterioration.

Fig.10

Fig.12

Carinii

pneumonia

165 respondents (multiple choice n=175)

165 respondents (multiple choice)

165 respondents Fig.11 Symptoms and frequency in the last year

No hospitalization

Hospitalization

(Number of

responses)

(Number of

Responses)

(Number of responses)

7

It was considered that

complications with a high

incidence may become serious

or chronic, the treatment of

which may be difficult, leading to

a major impact on daily life.

*Major answers are shown.

Susceptible to infection

Routine immunoglobulin replacement therapy

Stubborn infection

Infection may turn serious

Long-term intensive treatment (requiring regular hospital visits)

Must avoid crowds as much as possible

Susceptible to infections that healthy people do not develop

Must wear a mask when outdoors

Difficult to be engaged in energy-consuming activities

Must be vigilant for molds in general and

be cautious about the surrounding environment

Must not eat raw eggs, raw meat, or moldy foods (e.g., some cheeses)

Prone to pyrexia by air temperature changes

Strictly adhere to the prescription, such as medication including infection prophylaxis, G-CSF (gran), interferon-gamma, etc.

Must avoid touching soil

Arthralgia

Muddy or watery diarrhea > 3 times/day

Severe malaise and fatiguability requiring bed rest for > 1 hour/day

Do not approach roadworks and construction sites

Must avoid swimming in a public pool

Must avoid drinking unpasteurized water (including some PET bottled products)

Limit activities to light work like deskwork

Most avoid entering hot spring facilities

Frequent cleaning of air-conditioners required at home

Do not go out barefoot

May experience vomiting for > 2 times/day or nausea for >30 minutes/day

May have unexplained pyrexia for > 7 days/month (in total)

Must prevent bacterial growth by sterilizing fresh flowers

0% 10% 20% 30% 40% 50% 60%

Symptoms associated with susceptibility to infection ■ Treatment compliance ■ Restrictions on outings (to prevent infection)

Dietary restrictions (to prevent infection) ■ Activity restrictions (to prevent infection)

Fig.13 Chronic complications

Fig.14 the symptoms

165 respondents (multiple choice)

165 respondents (multiple choice n=1,015)

(Number of

responses)

(Number of

Responses)

8

Receive intensive (appropriate) treatment for infection as soon as possible to prevent deterioration

Wash hands and gargle carefully and frequently to avoid infection

Avoid crowds

Wear a mask when outdoors

Avoid getting close to molds in the surrounding

Avoid exhaustion as much as possible in various life situations to prevent pyrexia and opportunistic infections

Avoid eating raw eggs, raw meat, or moldy foods (e.g., some cheeses)

Receive intensive (appropriate) treatment and medication for suppuration in case of injury or dermatitis

Careful toothbrushing after each meal to prevent oral cavity infections, including periodontal disease and dental caries

Be cautious about temperature and carefully control room temperature to prevent pyrexia and infection.

Receive intensive (appropriate) treatment and medication for suppuration at surgery, including skin incisions and tooth extraction

Avoid swimming in a public pool or confirm the sanitization status before swimming

Vigilant for insect bites to prevent deterioration

Do not approach roadworks and construction sites to prevent Aspergillus infection. Avoid touching horticultural soil

Avoid drinking unpasteurized water (including some PET bottled products)

Protect with a mask against molds from air-conditioners. Clean home air-conditioners frequently

Avoid going out barefoot or touching soil

Avoid activities that exceed the deskwork level and take a break during activities

Protect against atopic dermatitis-like dermatitis caused by ultraviolet irradiation

PID can be life-threatening without lifelong treatment for the underlying disease and care for immune dysfunction.

In addition, PID patients live a life with limits depending on the type of PID. Understanding of the surroundings is

critical for PID patients to participate in society.

58.5% of the patients received routine immunoglobulin replacement therapy (injection) to prevent infection. Many

patients received oral medications, such as antibiotics and antifungals. As the half-life of immunoglobulin

products is three to four weeks, patients need to visit a medical institution regularly at least once a month.

Fig.15

Fig.16 Treatments for PID 159 respondents (multiple choice n=330)

3．Ongoing treatment

165 respondents (multiple choice, n=835)

(Number of

responses)

(Number of responses)

9

93 respondents 93 respondents

■ Measure trough

level of IgG

■ Not available

■ Know trough

level of IgG

■ I do not know

74.2% of the patients receiving routine replacement therapy with immunoglobulin products (IgG) knew their trough

levels of IgG, which suggested that physician's careful informed consent and patient's awareness of the disease

were in place. On the other hand, 12.9% of the patients answered that trough level of IgG was not measured, and

25.8% did not know their trough level, which suggested that some patients did not know it even though it was

measured.

mg/dL

■ 300 or less

■ 300～399

■ 400～499

■ 500～599

■ 600～699

■ 700～799

■ 800～899

■ 900～999

■ More than 1000

While the optimal trough level varies from

patient to patient, it is reported (in package

insert for IgG products) that the incidence of

infections and length of stay were 1.04

times/year and 0.70 days/year, respectively in

patients with trough level of 500 mg/dL or higher

IgG. In 66 patients who answered the trough

level, 7.5% had trough level of 500 mg/dL or

lower and 22.7% had trough level of 1,000 mg/dL

or higher. It seemed necessary to identify the

optimal trough level depending on the patient's

lifestyle changes while consulting with the

doctor.

0% 20% 40% 60% 80% 100%

■ All expenses are subsidized (excluding those under the upper limit of out-of-pocket payments) ■ Part of medical expenses for complication are not subsidized.

PID is designated as an intractable disease by the Japanese government and PID treatment costs, including those

for associated complications are subsidized. The survey respondents were asked if the medical expenses for

complications were subsidized or not, and it was found that 31.4% of the patients did not receive any subsidies. It

was considered necessary to promote understanding about the public assistance program among medical

institutions.

Fig.17 Measurement of trough level of serum IgG

Fig.20 Public assistance of treatment costs

Patients trough levels of serum IgG

66 respondents

137 respondents

Fig.18 Awareness of trough level of serum IgG

10

Treatment cost for PID is the burden

(155 respondents)

Treatment cost for complications is the burden

(151 respondents)

Transportation expenses for visiting a medical

institution is the burden

(152 respondents)

Medical expenses put pressure on cost of living

(151 respondents)

0% 20% 40% 60% 80% 100%

■ Very applicable ■ Slightly applicable ■ Less applicable ■ Not at all applicable

As mentioned above, there is a subsidy system for medical expenses for PID treatment, but patients need to bear

the costs under the cap of out-of-pocket payments. Even with subsidies, 49.1% of the patients (the sum of "very

applicable" and "slightly applicable") reported that the treatment cost was the burden. In addition, patients may

be required to pay treatment costs for complications or acute symptoms outside the subsidy system, and 34.5%

(the sum of "very applicable" and "slightly applicable") of the patients felt that such costs were the burden. Besides,

because many patients need to travel a long distance to a general hospital for regular treatment and treatment of

complications, these costs, including transportation expenses for the patient's family, seemed to be pressing on

their living expenses.

Kindergarten and nursery school

(122 respondents)

Elementary school

(115 respondents)

Junior high school

(85 respondents)

High school

(71 respondents)

0% 20% 40% 60% 80% 100%

■ 0 to 3 months ■ 4 to 6 months ■ 7 to 9 months ■ 10 to 12 months

64.3% to 70.4% of the patients were able to attend kindergarten, nursery school, elementary school,

junior high school, or high school for 10 to 12 months throughout the year. This may be because patients

had to be absent from kindergarten or school due to the necessity of regular visiting to a medical

institution or hospitalization for the treatment of PID and associated acute symptoms or complications.

In high schools, it is generally said that absence of 1/3 leads to holdover or that presence of more than 2/5 of the

actual number of school days is required. It was assumed that there were cases in which PID patients forced

themselves to attend school and were suffering from this.

Fig.21 Economic burden of treatment

9.0% 14.8% 9.8% 66.4%

7.0% 13.0% 15.7% 64.3%

8.2% 11.8% 10.6% 69.4%

9.9% 7.0% 12.7% 70.4%

4．Kindergarten and school life

12.3% 36.8% 25.2% 25.8%

14.6% 19.9% 29.1% 36.4%

22.4% 30.3% 28.9% 18.4%

12.6% 31.1% 30.5% 25.8%

Fig.22 How many months the patients attended school in a year

11

Fig.26 Compromise on career options because of the disease

Elementary school

(114 respondents)

Junior high school

(84 respondents)

High school

(72 respondents)

Elementary school

(115 respondents)

Junior high school

(84 respondents)

High school

(71 respondents)

■ Very important ■ Slightly important

■ Less important ■ Not at all important

■ Very important ■ Slightly important

■ Less important ■ Not at all important

The patients had a lot of limitations in their daily lives, and in school to comply with the instructions from a physician

and try to prevent infections. It can be assumed that it is very hard for growing children, even with the support

from their families. This is consistent with the afore mentioned influence on school days.

Elementary school

(114 respondents)

Junior high school

(84 respondents)

High school

(72 respondents)

Elementary school

(114 respondents)

Junior high school

(84 respondents)

High school

(72 respondents)

■ Very applicable ■ Slightly applicable

■ Less applicable ■ Not at all applicable

■ Very applicable ■ Slightly applicable

■ Less applicable ■ Not at all applicable

The number of the respondents who answered that the disease affected school class and performance increased

in upper grades and reached 36.1% in high school students (the sum of "very applicable" and "slightly applicable"),

and the impact on career options increased in upper grades to 31.9% in high school students. *Adult patients and patients who already graduated from kindergartens and schools also answered the questions based on the situation in

their school age.

Fig.24 Efforts to prevent infection as much as possible during school life

Fig.25 Absence from classes due to disease affected school performance

Fig.23 Compliance with instructions from physicians during school life

12

■ Employed as regular employees

■ Employed as non-regular employees

■ Not working

(Including employees on leave)

The employment rate of the patients aged 18

years and older was 59.0%. This was lower than

the 75.8%* employment rate of healthy general

people aged 15 to 64 years according to the

National Survey of the Ministry of Internal

Affairs and Communications. These findings

suggested that the patients were in a difficult

working environment. *Calculated based on December 2017 data from the Labor

Force Survey, Statistics Bureau, Ministry of Internal Affairs and

Communications

■ Previously employed but resigned or taking leave

■ Have never worked

■ Other

The reasons for not working were answered by

28 persons, and 42.9% of them answered that

they had worked before but resigned or were

taking leave due to some reasons. When asked

about the reason, 75% answered "because the

disease worsened."

■ I want to work, but I think it is

impossible considering the symptoms and conditions.

■ If possible, I would like to work with certain considerations for the disease at workplace.

■ I want to work even without considerations for the disease at workplace

■ Other

Twenty-five out of 29 (86.2%) of those who

were not working had intension to work. It is

much expected to have acceptance and

understanding at workplace about the disease,

including acute pyrexia and deterioration in

physical condition, in order for PID patients to

comply with restrictions and maintain their

physical condition while working. If physical

disability certificate is issued for PID,

understanding and support of society would

advance, and the number of PID patients in

employment would increase

5. Employment (*in the respondents aged 18 years and older)

Fig.28 Reasons for not working at present

Fig.29 Patient expectations about employment in the future

Fig.27 Current working conditions 83 respondents

13

Upper limb

Lower limb

165 respondents 12 respondents (multiple choice)

Yes No No answer

Twelve of the respondents (7.3%) were certified as physically handicapped, but it was not certified as a result of

PID-induced innate immune dysfunction.

*Fig.31 lists only major sites of dysfunction

Reduce the burden on medical expense (128 respondents)

A public transportation discount system

that helps patients when they see a specialist in far distance (125 respondents)

Employment framework for the physically handicapped expands the

range of their work like regular employment contract and jobs (124 respondents)

Able to work with appropriate considerations by the employment

framework for physically handicapped (126 respondents)

■ Very necessary ■ Somewhat necessary ■ Less necessary ■ Not at all necessary

It was found that approximately 80% of the patients considered that physical disability certification would help

reduce the economic burden of medical expenses and transportation expenses for the treatment of PID. About

80% of the patients expected that the certification would enable them to work in the employment framework for

the physically handicapped. Many PID patients showed expectation towards designation of physical disability

certification for immune dysfunction.

6. Physical disability certification

Fig.30 Patients with physical disability certification

(Patients)

Fig.32

Vision Trunk Heart Respiratory

(Number of responses)

14

Support for groups and networks of the same type of disease

Provision of the latest medical information

Supporting for the creation of networks between patients and their families

Advocacy to relevant organizations for designation of physical disability certification to for

immune dysfunction in PID

Medical lectures

Telephone consultation and e-mail consultation

Patient-family meetings

Medical consultations

■ Expect more frequent service ■ Sufficient at present ■ Expect less frequent service ■ I do not know well ■ No answer

The respondents expected various activities by Specified Nonprofit

Corporation, PID Tsubasa No Kai, where they looked forward to efforts to

encourage related organizations for designation of physical disability

certification for PIDs, to the dissemination of the latest medical information,

and to support the creation of networks among patients.

<Acknowledgement>

Blood Product Subcommittee of EFPIA, Japan extends deep gratitude to the patients and their families for their

participation in the questionnaire survey.

The Subcommittee also extends appreciation to Specified Nonprofit Corporation, PID Tsubasa No Kai, Shire

Japan K.K., Novo Nordisk Pharma K.K., and Bayer Yakuhin, Ltd. for their cooperation in the survey form

distribution and data aggregation.

April 2018.

Blood Product Subcommittee,

Biologics Committee

European Federation of

Pharmaceutical Industries and

Associations, Japan (EFPIA, Japan)

7. What the patients want for the society

ty Fig.33 Expectations to PID Tsubasa No Kai

15

Created in July 2018.